Human Immune System Humanized Modeling of Down Syndrome Immune Responses to Pluripotent Stem Cells

PROJECT NARRATIVE The proposed research is relevant to public health because it develops multiple biological material and data resources that could help overcome limitations of current pharmaceutical treatments and cell therapy efforts for a number of diseases that impact people with Down syndrome (DS), with the long-term potential to save lives as well as improve health-related quality-of-life. Further, it validates a novel strategy for achieving transplantation tolerance that will be applicable to a variety of future cell therapies in multiple disciplines, specifically enabling people with DS to access this new class of treatment. This research project is in line with key elements of NIH’s mission, as it focuses on reducing illness and disability.